Chidamide plus venetoclax as effective and safe maintenance therapy in high-risk acute myeloid leukemia and myelodysplastic syndrome post-transplantation

Abstract Relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the leading cause of treatment failure in high-risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), yet effective maintenance strategies are currently lacking. The combination of chidamide (CHI) and venetoclax (VEN) represents a novel approach. However, real-world data on its use are limited. In this study, eighteen high-risk transplant recipients received maintenance therapy with CHI (5 mg/day on days 1–7 or 1–14) plus VEN (100 mg on day 1, then 200 mg on days 2–7 or 2–14) every 2 months. Endpoints included overall survival (OS), disease-free survival (DFS), cumulative incidence of relapse (CIR), non-relapse mortality (NRM), and safety. After a median follow-up of 708 days, the 2-year OS and DFS were 73.9% and 56.6%, respectively. The 2-year CIR was 28.9%, and the 2-year NRM rate was 22.5%. The most common adverse events were hematologic, including neutropenia (13 episodes), thrombocytopenia (7), and anemia (7), all of which were manageable. No treatment-related mortality, tumor lysis syndrome, or severe acute graft-versus-host disease (GVHD) occurred. In conclusion, CHI plus VEN as post-transplant maintenance therapy shows encouraging efficacy and a manageable safety profile in high-risk AML/MDS patients, representing a promising strategy for relapse prevention.